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FDA Approves Genentech’s Hemlibra (Emicizumab-kxwh) for Hemophilia A with Inhibitors

First new medicine in nearly 20 years to treat people with hemophilia A with inhibitors

HEMLIBRA substantially reduced bleeds in adults and children

Only medicine that can be self-administered once weekly by injection under the skin (subcutaneously)

South San Francisco, CA -- November 16, 2017 --

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors. Nearly one in three people with severe hemophilia A can develop inhibitors to factor VIII replacement therapies, putting them at greater risk for life-threatening bleeds or repeated bleeds that can cause long-term joint damage. In two of the largest pivotal clinical studies for people with hemophilia A with inhibitors, HEMLIBRA was shown to substantially reduce bleeds in adults and children.

“People with hemophilia A who develop inhibitors face significant challenges preventing bleeds and typically require infusions of medicine multiple times a week, which can be especially difficult for young children and their families,” said Guy Young, M.D., director of Hemostasis and Thrombosis Program, Children’s Hospital Los Angeles, and professor of Pediatrics, University of Southern California Keck School of Medicine, Los Angeles, California. “This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week. This could make a meaningful difference for these children.”

“Before HEMLIBRA, my 7-year-old son needed intravenous infusions that could take up to two hours at least three times a week, so our lives revolved around his treatment,” said Amber Hill, mother of a young boy with hemophilia A with inhibitors. “With HEMLIBRA, he now has an injection once a week that he has proudly learned to administer himself to help prevent bleeds. Not only has he had fewer bleeds compared to his prior treatment, he has more time to be a kid and we have more quality time as a family because of the new treatment schedule.”

In the Phase III HAVEN 1 study, people 12 years of age or older with hemophilia A with inhibitors who received HEMLIBRA prophylaxis had a statistically significant reduction in treated bleeds of 87 percent (95 percent CI: 72.3; 94.3, p<0.0001) compared to those who received no prophylaxis. In a first-of-its-kind intra-patient analysis, HEMLIBRA prophylaxis resulted in a statistically significant reduction in treated bleeds of 79 percent (95 percent CI: 51.4; 91.1, p=0.0003) compared to previous treatment with bypassing agent (BPA) prophylaxis collected in a non-interventional study (NIS) prior to enrollment.

Interim results from the pivotal HAVEN 2 study in children younger than 12 years of age with hemophilia A with inhibitors showed that 87 percent (95 percent CI: 66.4; 97.2) of children who received HEMLIBRA prophylaxis experienced zero treated bleeds. In an intra-patient analysis of 13 children who had participated in the NIS, HEMLIBRA prophylaxis resulted in a 99 percent reduction in treated bleeds compared to previous treatment with a BPA either as prophylaxis (n=12) or on-demand (n=1). The most common adverse events (AEs) occurring in 10 percent or more of people treated with HEMLIBRA in pooled studies were injection site reactions, headache and joint pain (arthralgia).

“Today’s approval of HEMLIBRA represents an important advancement for people with hemophilia A with inhibitors, who have struggled to manage their bleeding disorder and haven’t had a new medicine in nearly 20 years,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We believe HEMLIBRA will improve protection against bleeds and reduce the treatment administration burden for people with hemophilia A with inhibitors, and we are committed to helping them access this medicine.”

HEMLIBRA will be available to people in the U.S. shortly after approval. Genentech is committed to helping people with hemophilia A with inhibitors access HEMLIBRA and will be offering comprehensive services to help minimize barriers to access and reimbursement. Patients can call (866) HEMLIBRA (436-5427) for more information. For people who qualify, Genentech also plans to offer patient assistance programs through Genentech Access Solutions. More information is available at (866) 4ACCESS/(866) 422-2377 or http://www.Genentech-Access.com .

HEMLIBRA was reviewed by the FDA under Priority Review and granted Breakthrough Therapy Designation by the FDA in people 12 years of age or older with hemophilia A with inhibitors in September 2015. Data from HAVEN 1 and HAVEN 2 are being reviewed under accelerated assessment by the European Medicines Agency (EMA) and submissions to health authorities around the world are ongoing.

HEMLIBRA is being studied in a robust clinical development program that includes two additional Phase III studies. HAVEN 3 is evaluating HEMLIBRA prophylaxis dosed once weekly or once every other week in people 12 years of age or older with hemophilia A without inhibitors to factor VIII. HAVEN 4 is evaluating HEMLIBRA prophylaxis dosed every four weeks in people 12 years of age or older with hemophilia A with or without inhibitors.

About HAVEN 1 (NCT02622321)

HAVEN 1 is a randomized, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of once-weekly subcutaneous administration of HEMLIBRA prophylaxis compared to no prophylaxis in adults and adolescents with hemophilia A with inhibitors to factor VIII. The study included 109 patients (12 years of age and older) with hemophilia A with inhibitors to factor VIII, who were previously treated with BPAs on-demand or as prophylaxis. Patients previously treated with on-demand BPAs were randomized in a 2:1 ratio to receive HEMLIBRA prophylaxis (Arm A) or no prophylaxis (Arm B). Patients previously treated with BPAs as prophylaxis received HEMLIBRA prophylaxis (Arm C). Additional patients previously treated with on-demand BPAs were also enrolled in a separate arm (Arm D). On-demand treatment of breakthrough bleeds with BPAs was allowed per protocol in all arms.

An improvement in Physical Health Score of the Haemophilia-specific Quality of Life (Haem-A-QoL) questionnaire was observed with HEMLIBRA prophylaxis compared to no prophylaxis. This was measured at 25 weeks in adults 18 years of age and older and evaluated hemophilia-related symptoms (painful swellings and presence of joint pain) and physical function (pain with movement and difficulty walking far).

HEMLIBRA may cause serious side effects when used with aPCC (FEIBA ®), including thrombotic microangiopathy (TMA) and blood clots (thrombotic events). Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis. As previously reported, three people experienced TMA events and two people experienced serious thrombotic events in the HAVEN 1 study.

About HAVEN 2 (NCT02795767)

HAVEN 2 is a single-arm, multicenter, open-label, clinical study in children younger than 12 years of age with hemophilia A with inhibitors to factor VIII. The study is evaluating the efficacy, safety and pharmacokinetics of once-weekly subcutaneous administration of HEMLIBRA prophylaxis. The interim efficacy analysis, after at least 12 weeks of treatment, included 23 children.

After a median observation time of 38.1 weeks, the interim analysis showed that 87 percent (95 percent CI: 66.4; 97.2) of children who received HEMLIBRA prophylaxis experienced zero treated bleeds. Interim data also showed:

34.8 percent (95 percent CI: 16.4; 57.3) of children experienced zero bleeds overall, which includes all treated and non-treated bleeds.

In an intra-patient analysis, 13 children who had participated in the NIS had an annualized bleeding rate (ABR) for treated bleeds of 17.2 (95 percent CI: 12.4; 23.8) on previous treatment with a BPA either as prophylaxis (n=12) or on-demand (n=1) compared to 0.2 (95 percent CI: 0.1; 0.8) on HEMLIBRA prophylaxis, corresponding to a 99 percent reduction in bleed rate. On HEMLIBRA prophylaxis, 11 children (84.6 percent) experienced zero treated bleeds.

The most common AEs occurring in 10 percent or more of people treated with HEMLIBRA in pooled studies were injection site reactions, headache and joint pain (arthralgia).

About HEMLIBRA

HEMLIBRA is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients. HEMLIBRA is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once weekly. HEMLIBRA was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed by Chugai, Roche and Genentech.

HEMLIBRA U.S. Indication

HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.

Important Safety Information

What is the most important information to know about HEMLIBRA?

HEMLIBRA increases the potential for blood to clot. Discontinue prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. Carefully follow the healthcare provider’s instructions regarding when to use an on-demand bypassing agent, and the dose and schedule one should use.

HEMLIBRA may cause the following serious side effects when used with aPCC (FEIBA®), including:

Thrombotic microangiopathy (TMA). This is a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs. Patients should get medical help right away if they have any of the following signs or symptoms during or after treatment with HEMLIBRA:

confusion

weakness

swelling of arms and legs

yellowing of skin and eyes

stomach (abdomen) or back pain

nausea or vomiting

feeling sick

decreased urination

Blood clots (thrombotic events). Blood clots may form in blood vessels in one’s arm, leg, lung or head. Patients should get medical help right away if they have any of these signs or symptoms of blood clots during or after treatment with HEMLIBRA:

swelling in arms or legs

pain or redness in the arms or legs

shortness of breath

chest pain or tightness

fast heart rate

cough up blood

feel faint

headache

numbness in the face

eye pain or swelling

trouble seeing

If aPCC (FEIBA®) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (FEIBA®) total.

How should patients use HEMLIBRA?

HEMLIBRA may interfere with laboratory tests that measure how well blood is clotting and may cause a false reading. Patients should talk to their healthcare provider about how this may affect their care.

What are the other possible side effects of HEMLIBRA?

The most common side effects of HEMLIBRA include: redness, tenderness, warmth, or itching at the site of injection; headache; and joint pain.

These are not all of the possible side effects of HEMLIBRA. Patients should call their doctor for medical advice about side effects.

Side effects may be reported to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Side effects may also be reported to Genentech at (888) 835-2555.

Hemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Hemophilia affects around 20,000 people in the United States, with hemophilia A being the most common form and approximately 50-60 percent of people living with a severe form of the disorder.

People with hemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with hemophilia A can bleed frequently, especially into their joints or muscles. These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage.

A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding. Most people with hemophilia A who develop inhibitors typically infuse BPA therapies, either on-demand (episodic) or as prophylaxis.

About Genentech in hemophilia

In 1984, Genentech scientists were the first to clone recombinant factor VIII in response to the contaminated hemophilia blood supply crisis of the early 1980s. For more than 20 years, Genentech has been developing medicines to bring innovative treatment options to people with diseases of the blood within oncology, and in hemophilia A. Genentech is committed to improving treatment and care in the hemophilia community by delivering meaningful science and clinical expertise. For more information visit http://www.gene.com/hemophilia .

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.